Oral Factor D Inhibition Falls Short in AChR-Positive Generalized Myasthenia Gravis: What the Vemircopan Phase 2 Trial Means for Complement-Targeted Therapy

Oral Factor D Inhibition Falls Short in AChR-Positive Generalized Myasthenia Gravis: What the Vemircopan Phase 2 Trial Means for Complement-Targeted Therapy

In a phase 2 randomized trial, oral vemircopan did not improve clinical outcomes over placebo in AChR-antibody–positive generalized myasthenia gravis, despite a mechanistically attractive strategy to selectively inhibit the alternative complement pathway.